Clinical trials: Phase 1, open to volunteers
Renal Failure Congenital, Congenital Renal Anomaly Nos
Serial Amnioinfusions as Regenerative Therapy for Pulmonary Hypoplasia
Congenital lower urinary tract obstruction (LUTO) is a heterogeneous group of congenital anomalies that lead bladder outlet obstruction.
- Conditions
- Renal Failure Congenital, Congenital Renal Anomaly Nos, Renal Agenesis and Dysgenesis, Lower Urinary Tract Obstructive Syndrome
- Trial name
- NCT03723564
- Testing
- Lactated Ringers Solution for Injection
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Mayo Clinic
Myelodysplastic Syndromes
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults.
- Trial name
- NCT06303193
- Testing
- pacritinib
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- National Cancer Institute (NCI)
Locally Advanced Solid Tumor, Metastatic Solid Tumor
A Study of Neladalkib (NVL-655) in Patients With Advanced NSCLC and Other Solid Tumors Harboring ALK Rearrangement or Activating ALK Mutation (ALKOVE-1)
Phase 1/2, dose escalation and expansion study designed to evaluate the safety and tolerability of neladalkib (NVL-655), determine the recommended phase 2 dose (RP2D), and evaluate the antitumor activity in patients with advanced…
- Trial name
- NCT05384626
- Testing
- Neladalkib (NVL-655), Midazolam, Repaglinide
- Where
- 74 sites in United States, Australia and 13 more
- Volunteers
- 840 people
- Sponsor
- Nuvalent Inc.
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
This phase I/II trial studies the side effects and best dose of iberdomide when given together with chemoimmunotherapy drugs and to see how well it works in treating patients with neuroblastoma that has come back after a period…
- Conditions
- Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma, Refractory Ganglioneuroblastoma, Refractory Neuroblastoma
- Trial name
- NCT07437963
- Testing
- Bone Marrow Aspiration, Bone Marrow Biopsy, Computed Tomography
- Where
- 4 sites in United States
- Volunteers
- 76 people
- Sponsor
- National Cancer Institute (NCI)
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Background: Blood cancers (such as leukemias or lymphomas) often do not respond to standard treatments. A transplant of blood stem cells from a healthy donor can help people with these cancers.
- Trial name
- NCT05436418
- Testing
- Melphalan, Sirolimus, Total Body Irradiation (TBI)
- Where
- 2 sites in United States
- Volunteers
- 260 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Lymphoma, CLL
A Phase I Dose Finding Study of MB-CART2219.1
A Phase I dose finding study of MB-CART2219.1 targeting CD19/CD22 in adult and pediatric patients with relapsed/refractory B-cell malignancies
- Conditions
- Lymphoma, CLL, Acute Lymphoblastic Leukemia, Pediatric
- Trial name
- NCT07108868
- Testing
- CAR-T cells targeting CD19 and CD22
- Where
- 2 sites in Germany
- Volunteers
- 36 people
- Sponsor
- University Hospital Tuebingen
Hyper IgE Syndrome From STAT3 Mutation, Job s Syndrome
Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis
Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system.
- Conditions
- Hyper IgE Syndrome From STAT3 Mutation, Job s Syndrome, HIES, Lupus
- Trial name
- NCT07262983
- Testing
- baricitinib
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Obstructive Sleep Apnea
DRonabinol Treatment of OSA
This is an open-label trial that tests the effectiveness of Dronabinol in treating obstructive sleep apnea (OSA). All participants will take oral Dronabinol for two weeks.
- Trial name
- DROSA (NCT06477952)
- Testing
- Dronabinol
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- VA Office of Research and Development
X-linked Severe Combined Immunodeficiency (XSCID)
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
This Phase I/II clinical trial will study the safety and potential benefits of gene therapy for up to 35 people aged 2 to 50 with X-linked severe combined immunodeficiency (XSCID), a rare genetic condition that makes it difficult…
- Trial name
- NCT01306019
- Testing
- Campath, Palifermin, Busulfan
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hemophilia A
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
WP44714 is a Phase I/II, open-label, non-randomized, global, multicenter trial consisting of two parts: Part 1 is a multiple-ascending dose (MAD) study in adult and adolescent male participants with severe or moderate hemophilia…
- Trial name
- NCT05987449
- Testing
- NXT007
- Where
- 14 sites in United States, Canada and 4 more
- Volunteers
- 60 people
- Sponsor
- Hoffmann-La Roche
Mature B-Cell Non-Hodgkin Lymphoma
A Study to Evaluate Glofitamab Monotherapy and Glofitamab + Chemoimmunotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Mature B-Cell Non-Hodgkin Lymphoma
The purpose of this study is to evaluate the safety and efficacy of glofitamab, as monotherapy and in combination with a standard chemoimmunotherapy regimen: rituximab, ifosfamide, carboplatin, and etoposide (R-ICE) in pediatric…
- Trial name
- iMATRIX GLO (NCT05533775)
- Testing
- Obinutuzumab, Glofitamab, Rituximab
- Where
- 30 sites in United States, Australia and 11 more
- Volunteers
- 65 people
- Sponsor
- Hoffmann-La Roche
Ebola-Bundibugyo Virus Disease (Ebola-BDBV)
A Study to Investigate the Safety and Immunogenicity of mRNA-1469 in African Participants 1 to 65 Years of Age
The main objective of this trial is to evaluate the safety of mRNA-1469
- Trial name
- NCT07919197
- Testing
- mRNA-1469
- Where
- Uganda
- Volunteers
- 224 people, healthy volunteers welcome
- Sponsor
- ModernaTX, Inc.
Refractory Ewing Sarcoma, Relapsed Ewing Sarcoma
Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma
This study is conducted in two phases. The phase 1 portion of the study evaluates the safety, tolerability, pharmacokinetics (PK), recommended phase 2 dose (RP2D), and effectiveness of lurbinectedin monotherapy in pediatric…
- Conditions
- Refractory Ewing Sarcoma, Relapsed Ewing Sarcoma, Ewing Sarcoma
- Trial name
- NCT05734066
- Testing
- Lurbinectedin
- Where
- 16 sites in United States, Canada
- Volunteers
- 60 people
- Sponsor
- Jazz Pharmaceuticals
Seasonal Influenza
A Study to Evaluate the Safety, Reactogenicity, and Immunogenicity of mRNA-1010 in Healthy Children
The purpose of this study is to evaluate safety, reactogenicity, and immunogenicity of mRNA-1010 in healthy children (9 to <18 years of age).
- Trial name
- NCT07870330
- Testing
- mRNA-1010, Fluarix Trivalent
- Where
- 6 sites in United States
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- ModernaTX, Inc.
Hematologic Diseases
Bispecific CD19/CD20-Targeted Chimeric Antigen Receptor (CAR) Modified T Cells With 4-1BB and Mutated CD28 Costimulatory Domains in Patients With Relapsed or Refractory CD19+ Hematologic Malignancies
To determine the safety, toxicity, and maximum tolerated dose (MTD) of CD19/CD20 bispecific CAR-T cells with 4-1BB and mutated CD28 costimulatory domains (20-19 Tan BB06z) in patients with relapsed or refractory CD19+ aggressive…
- Trial name
- NCT07701889
- Testing
- 20-19 Tan BB06oz CAR T
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Roswell Park Cancer Institute
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
The goal of this clinical trial is to learn whether a single dose of buccal buprenorphine (BELBUCA) is safe when used in place and/or with full agonist opioids that would otherwise be started during a sickle cell pain crisis…
- Conditions
- Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children
- Trial name
- NCT07799571
- Testing
- Buprenorphine Buccal Film [Belbuca]
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Natasha Archer
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
A Phase I open-label, multicenter study, to evaluate the safety, feasibility, and maximum tolerated dose (MTD) of treating children with newly diagnosed DIPG or recurrent neuroblastoma with molecular targeted therapy in…
- Trial name
- PEACH (NCT04837547)
- Testing
- Tumor-specific ex vivo expanded autologous lymphocyte transfer (TTRNA-xALT)
- Where
- 3 sites in United States
- Volunteers
- 24 people
- Sponsor
- University of Florida
Viral Infection
TETRAVI Multivirus CTL for Treatment of EBV, CMV, Adenovirus, and BK Infections Post Allogeneic SCT.
The purpose of this study is to use VSTs (virus-specific T cells) from a donor that is a partial HLA (human leukocyte antigen) match with the patient to treat viral infections after an allogeneic hematopoietic stem cell…
- Trial name
- TETRAVI (NCT04013802)
- Testing
- HLA-matched VSTs
- Where
- 2 sites in United States
- Volunteers
- 47 people
- Sponsor
- Baylor College of Medicine
Graft Versus Host Disease (cGvHD)
Ex Vivo-Expanded Regulatory T Cells Plus Low-Dose Interleukin-2 for Steroid-Refractory Chronic GVHD
Hematopoietic Stem Cell Transplant (HCT) has been used to treat children and adults who have leukemia, lymphoma and other cancers of blood and immune cells since the 1970s. For many of these forms of cancer, HCT works well.
- Trial name
- EVENT (NCT06991361)
- Testing
- EVE-Treg
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Sarah Nikiforow, MD
Acute Leukemia
A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2D[s]) of bleximenib in phase 1 Part 1 (Dose Escalation) and to determine the safety and tolerability at RP2D in Phase 1 Part 2 (Dose expansion).
- Trial name
- cAMeLot-1 (NCT04811560)
- Testing
- Bleximenib
- Where
- 102 sites in United States, Australia and 11 more
- Volunteers
- 420 people
- Sponsor
- Janssen Research & Development, LLC